MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Institute of Health and Society, Newcastle University, Newcastle upon Tyne, UK.
Stat Med. 2019 Aug 15;38(18):3305-3321. doi: 10.1002/sim.8194. Epub 2019 May 21.
Multiarm clinical trials, which compare several experimental treatments against control, are frequently recommended due to their efficiency gain. In practise, all potential treatments may not be ready to be tested in a phase II/III trial at the same time. It has become appealing to allow new treatment arms to be added into on-going clinical trials using a "platform" trial approach. To the best of our knowledge, many aspects of when to add arms to an existing trial have not been explored in the literature. Most works on adding arm(s) assume that a new arm is opened whenever a new treatment becomes available. This strategy may prolong the overall duration of a study or cause reduction in marginal power for each hypothesis if the adaptation is not well accommodated. Within a two-stage trial setting, we propose a decision-theoretic framework to investigate when to add or not to add a new treatment arm based on the observed stage one treatment responses. To account for different prospect of multiarm studies, we define utility in two different ways; one for a trial that aims to maximise the number of rejected hypotheses; the other for a trial that would declare a success when at least one hypothesis is rejected from the study. Our framework shows that it is not always optimal to add a new treatment arm to an existing trial. We illustrate a case study by considering a completed trial on knee osteoarthritis.
多臂临床试验常因效率增益而被推荐,其比较了几种实验治疗方法与对照治疗方法。但在实践中,并非所有潜在的治疗方法都能同时准备好进入 II/III 期临床试验。使用“平台”试验方法允许新的治疗组加入正在进行的临床试验,这种方法变得很有吸引力。据我们所知,在文献中尚未探讨何时向现有试验中添加臂的许多方面。大多数关于添加臂的工作都假设只要有新的治疗方法可用,就会开放新的臂。如果适应不良,这种策略可能会延长研究的总持续时间,或导致每个假设的边际效力降低。在两阶段试验设置中,我们提出了一种基于观察到的第一阶段治疗反应的决策理论框架,以研究何时添加或不添加新的治疗臂。为了考虑多臂研究的不同前景,我们以两种不同的方式定义效用;一种用于旨在最大化拒绝假设数量的试验;另一种用于当研究中至少有一个假设被拒绝时宣布成功的试验。我们的框架表明,向现有试验中添加新的治疗臂并不总是最优的。我们通过考虑一个已完成的膝关节骨关节炎试验来说明一个案例研究。